AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models.

Nat Commun · 2025 · PMC11880196 · PMID 40038304

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highly significantno p-value reported

The dose of 3E13 vg/kg yielded robust and highly significant improvements with decreased hypertrophy of 5.3 ± 0.8 mg/g LV mass and improved EF of 26 ± 3.0%.

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